Gene therapy with etranacogene dezaparvovec is successful in the treatment of haemophilia B, according to an international study, in which Erasmus MC also participated. The results recently appeared in The New England Journal of Medicine.
Patients with haemophilia B produce less coagulation factor IX due to a DNA mutation. Gene therapy can change this. A healthy factor IX gene is introduced into the patient’s body, which ensures the production of coagulation factor IX.
The therapy activates production after just 1 treatment. The first research results are positive: at Erasmus MC, 6 patients have now been successfully treated with etranacogene dezaparvovec in a study context.
Factor IX was produced in all patients and bleeding was reduced. As a result, they no longer had to administer coagulation factors twice a week. Their quality of life also improved.
It is not certain how long the therapy will work. Some patients have not received coagulation factor for 6 years. It remains to be seen what the long-term effects will be, but for now the production of factor IX seems stable over time.
It has recently been announced that the European Medicines Agency (EMA) has approved this gene therapy for haemophilia B.
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